Biogen and Ionis Pharmaceuticals announced that Biogen exercised its option to obtain from Ionis a worldwide, exclusive, royalty-bearing license to develop and commercialize BIIB067 (IONIS-SOD1RX), an investigational treatment for amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1) mutations.

ALS with SOD1 mutations is a subtype of familial ALS and accounts for approximately two percent of all ALS cases.

“Biogen and Ionis have a shared goal of bringing innovative therapies to those living with severe neurological diseases where there is significant unmet medical need,” said Michael Ehlers, M.D., Ph.D., executive vice president, research and development at Biogen. “The progress made to date in the BIIB067 clinical program is a positive step forward. We are committed to our goal of bringing forward a therapy for patients with ALS with SOD1 mutations, who currently have limited or no treatment options.”

The decision to exercise the option was based on a positive interim analysis of a randomized, placebo controlled single- and multiple-ascending dose Phase 1 study (n=70) that achieved proof-of-biology and proof-of-concept for BIIB067. At the highest dose tested (n=10), treatment with BIIB067 over a three month period resulted in a statistically significant lowering of SOD1 protein levels in the cerebrospinal fluid (p=0.002) and a numerical trend towards slowing of clinical decline as measured by the ALS Functional Rating Scale-Revised, both compared to placebo (n=12). The safety and tolerability profile in this analysis supports continued development of BIIB067 in ALS. Based on these results, Biogen plans to advance BIIB067 to a pivotal clinical study.

“The results from this study provide hope to SOD1 ALS patients who are suffering with this devastating disease,” said C. Frank Bennett, Ph.D., senior vice president of research and franchise leader for neurological programs at Ionis. “BIIB067 is the first investigational medicine targeting the known cause of this familial form of ALS to advance towards a pivotal study. Our broad strategic collaborations with Biogen have produced not only SPINRAZA, but six more drugs moving forward in development and a large number of preclinical and research programs. We believe our proprietary antisense technology will continue to bring benefit to patients now and in the future.”

As a part of the option exercise, Biogen made a one-time $35 million payment to Ionis. Future payments may include potential post-licensing milestone payments of up to $55 million and royalties in the low to mid-teen percentages on annual worldwide net sales. Biogen will be solely responsible for the costs and expenses related to the development, manufacturing and commercialization of BIIB067 following the option exercise.

ALS is a rare, fatal neurodegenerative disease characterized by motor neuron loss in the brain and spinal cord that are responsible for controlling voluntary muscle movement. Symptoms may vary depending on the location of the motor neuron failure and may include muscle stiffness and spasticity or spontaneous muscle twitching followed by atrophy. The average life expectancy for an ALS patient is less than five years from the time of diagnosis. Mutations within multiple genes are believed to cause ALS, including mutations in the SOD1 gene.

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